Berlin Heart Device Provides Life-Saving "Bridge" for Young Children

A new study brings hope for hundreds of babies born with heart failure each year. The study, led by Texas Children's Hospital and Baylor College of Medicine (BCM), projected how a tiny heart pump that maintains blood flow in babies and small children with serious heart failure, proved to be effective and lifesaving.

This study mainly focused on the safety and probable benefit of the Berlin Heart EXCOR Pediatric Ventricular Assist Device (VAD), the only VAD available for babies and children. The researchers noticed that patients who received the Berlin Heart, survived for a longer period without many problems and were more likely to receive a transplant or recover heart function, than children who were maintained on more traditional support using extracorporeal membrane oxygenation (ECMO).

"This study is unprecedented and represents broad collaboration among the top pediatric cardiac transplantation centers in North America, really the who's who in the field," said Dr. Charles Fraser Jr., surgeon-in-chief at Texas Children's Hospital and professor of Surgery and Pediatrics at BCM. Fraser is also the corresponding author of the report and was national principal investigator of the study. "This study is now the gold standard for VAD therapies in children. Everything going forward will be compared to this."

Prior to the Berlin Heart, physicians opted for more complicated medical therapies in order to treat children with heart failure.  Newborns and small children often died as they waited, since no more than 70 or 80 small donor hearts become available each year. ECMO provided only short-term support when the child's heart failed completely. The Berlin Heart offers families a new ray of hope.

"With the Berlin Heart, we have a more effective therapy to offer patients earlier in the management of their heart failure," said Fraser. "When we sit with parents, we have real data to offer so they can make an informed decision. This is a giant step forward."

What's remarkable is that children who are hooked to this device are permitted to leave the ICU and shift to a normal hospital room, where they receive rehabilitation and better nutrition while they wait for a transplant.

"This prospective trial is unprecedented because it was not a look back at how children on the device had fared but instead followed their clinical course from the device's implantation. The results were compared to those of children who received ECMO, the only other method of treatment," said Fraser, also chief of congenital heart surgery at Texas Children's.

"We thought we knew a lot about this device and how it worked, but the FDA was insistent that we conduct a trial and they were right. We did not know as much as we should about how the device performs in practice."

The Berlin Heart is not fully implanted inside the body. Physicians insert cannulas, or flexible tubes, in the heart and they extend through the skin and connect to a small pump located outside the body. That pump, along with its computerized drive unit, maintains blood flow.

The study enrolled 48 children who received the device at 17 hospitals in the US and Canada between 2007 and 2010. It compared the outcomes of the participants, from infancy to 16 years of age and divided them into two groups based on body size, with pediatric matches in a national registry of patients who received temporary support from a heart-lung machine, such as that used in open heart surgery. 92 percent of the larger children, average age 9, were successfully transplanted or weaned off the Berlin Heart at 192 days. By contrast, 33 percent of same-sized children on a heart-lung machine had died at 30 days.

Among the smaller children, average age 1, 88 percent were successfully transplanted or weaned off the Berlin Heart at 174 days. 25 percent of the comparison group had died at 21 days.

The study did find a higher-than-anticipated rate of stroke associated with the device - 29 percent - though Fraser said that all children recovered without any neurological impairment. It also found significant amounts of bleeding and infection, both easily controlled.

"I don't think we are far from a small, implantable VAD in children," Fraser said. "The implantable devices will be less cumbersome. There are approved, fully implantable devices already available for adults. This gives us the drive to push even further for more devices like this that can be life-saving options for these children who have no other treatment options."